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4 results

  • Effectiveness and safety of caplacizumab in acquired thrombotic thrombocytopenic purpura: health technology assessment and classification according to the methodology established in Colombia

  • Jahir A. Soto-Mora, Lina M. Gómez-Espitia, Pieralessandro Lasalvia, Camilo A. Castellanos Moreno, Carol A. Casallas Vanegas, Sergio A. Londoño Gutiérrez
  • Journal:
    International Journal of Technology Assessment in Health Care / Volume 39 / Issue 1 / 2023
    Published online by Cambridge University Press:
    21 July 2023, e48
    • Article
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  • Objectives

    Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare hematological disease whose clinical management includes caplacizumab along with plasma exchange and immunosuppression, according to international guidelines. Caplacizumab has been available in Colombia since 2022. This study seeks to determine the therapeutic classification of caplacizumab according to the methodology of the Instituto de Evaluación Tecnológica en Salud.

    Methods

    The classification was carried out through a deliberative process following the modified Delphi technique, with a panel of experts, made up of four hemato-oncologists, a pharmaceutical chemist, and a patient. The results of effectiveness and safety obtained through a systematic review, therapeutic thresholds (clinical significance), and degree of acceptability (willingness to use the technology) were used for the classification.

    Results

    Fourteen effectiveness and safety outcomes were submitted for the classification process. Caplacizumab showed clinical significance for some effectiveness outcomes, was not considered inferior in terms of safety, and displayed acceptability of use. Through consensus, the panel determined that caplacizumab plus the standard regimen is superior to the standard regimen in terms of treatment response and composite outcome, and no different for the other effectiveness and safety outcomes. Likewise, in overall terms, the panel determined that caplacizumab together with the standard regimen is superior to the standard regimen.

    Conclusion

    Treatment with caplacizumab together with the standard regimen was considered superior to the standard regimen for the treatment of patients with aTTP, as it showed clinically significant benefits in critical outcomes for decision making, and a safety profile no different to its comparator.

  • 66 - Overdoses

  • from Section 11 - Environmental emergencies
  • Edited by Kaushal Shah, Jarone Lee, Kamal Medlej, American University of Beirut, Scott D. Weingart
  • Book:
    Practical Emergency Resuscitation and Critical Care
    Published online:
    05 November 2013
    Print publication:
    24 October 2013, pp 431-450

  • Summary

    This chapter discusses the diagnosis, evaluation and management of disseminated intravascular coagulation and thrombotic thrombocytopenic purpura (TTP)/hemolytic uremic syndrome (HUS). The classic presentation of TTP involves a pentad of symptoms that include fever, neurological signs, anemia, thrombocytopenia, and renal dysfunction. This collection of symptoms is only seen in 20-30% of cases and it is strongly recommended to suspect the condition and manage it as such if a patient exhibits three or more of those features. HUS is most commonly seen in children and often follows an infectious illness, usually diarrhea. Morbidity and mortality in patients with TTP/HUS are usually attributed to thrombosis rather than anemia and bleeding. Patients with TTP can present with neurological symptoms that can be life threatening themselves or complicated by a life-threatening event. Patients (usually children) presenting with HUS may have significant renal dysfunction requiring dialysis.

  • Chapter 20 - Nutrition

  • from Section 3 - Special critical care tools and techniques
  • Edited by Marc van de Velde, Helen Scholefield, Lauren A. Plante
  • Book:
    Maternal Critical Care
    Published online:
    05 July 2013
    Print publication:
    04 July 2013, pp 203-216

  • Summary

    This chapter focuses on three entities namely disseminated intravascular coagulation (DIC), HELLP syndrome, and thrombotic thrombocytopenic purpura (TTP), which represents unique and critical threats to the well-being of mother and fetus during peripartum period. It is concerned with the etiology, clinical features, diagnostic methods and management of these entities. In non-bleeding patients with DIC, platelets and factor replacement should not be administered prophylactically or based on laboratory tests alone. The treatment of HELLP involves monitoring and responding to maternal signs and symptoms, particularly when pre-eclampsia is present, and includes fluid management and the use of antihypertensive agents and magnesium sulfate for seizure prophylaxis. Plasma exchange is the treatment of choice for TTP. The optimal treatment regimen for obstetric coagulation disorders continues to evolve, given the frequently dynamic clinical situation, the presence and health of the fetus, and a growing interest in conducting investigations during the peripartum period.

  • 51 - KOHLMEIER-DEGOS’ DISEASE (MALIGNANT ATROPHIC PAPULOSIS)

  • from PART V: - SYSTEMIC DISORDERS THAT ALSO INVOLVE THE CEREBROVASCULAR SYSTEM
  • Edited by Louis R. Caplan, Julien Bogousslavsky
  • Book:
    Uncommon Causes of Stroke
    Published online:
    06 January 2010
    Print publication:
    09 October 2008, pp 377-380

  • Summary

    The underlying pathological mechanism of thrombotic thrombocytopenic purpura (TTP) is the presence of microvascular thrombi that partially occlude the vascular lumins with overlying proliferative endothelial cells. Coombs-negative hemolytic anemia and severe thrombocytopenia owing to platelet clumping in the microcirculation are the most outstanding laboratory abnormalities. Classically, TTP has been recognized by the pentad of microangiopathic hemolytic anemia, thrombocytopenia, neurological symptoms, fever, and renal involvement, though only 20 to 40 percentage of patients will manifest the classic pentad. TTP remains a life-threatening disease the mortality rate of which may be as high as 90 percentage when untreated. Diagnosis is mainly based on hematological findings and a broad variety of neurological abnormalities, including ischemic or, less often, hemorrhagic stroke. Plasma exchange (PE) is currently the mainstay of treatment; however, rapid advances in the understanding of TTP pathophysiology may offer more specific and effective therapies in the near future.